MX2021009388A - Treatment of ciliopathies. - Google Patents

Treatment of ciliopathies.

Info

Publication number
MX2021009388A
MX2021009388A MX2021009388A MX2021009388A MX2021009388A MX 2021009388 A MX2021009388 A MX 2021009388A MX 2021009388 A MX2021009388 A MX 2021009388A MX 2021009388 A MX2021009388 A MX 2021009388A MX 2021009388 A MX2021009388 A MX 2021009388A
Authority
MX
Mexico
Prior art keywords
polyribonucleotide
ciliopathy
subject
ciliogenesis
present disclosure
Prior art date
Application number
MX2021009388A
Other languages
Spanish (es)
Inventor
Carsten Rudolph
Christian Dohmen
Johannes Geiger
Verena Kretzschmann
Rebekka Kubisch-Dohmen
Manish Aneja
Ludwig Weiss
Heymut Omran
Petra Pennekamp
Kai Wohlgemuth
Sandra Cindric
Niki Tomas Loges
Johanna Raidt
Steege Adrian Ter
Original Assignee
Ethris Gmbh
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ethris Gmbh filed Critical Ethris Gmbh
Publication of MX2021009388A publication Critical patent/MX2021009388A/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
    • A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
    • A61K31/198—Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0043—Nose

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pulmonology (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Biomedical Technology (AREA)
  • Otolaryngology (AREA)
  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Dispersion Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Analytical Chemistry (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Microbiology (AREA)
  • Tropical Medicine & Parasitology (AREA)

Abstract

The present disclosure provides a pharmaceutical composition comprising a polyribonucleotide for use in treating a ciliopathy in a subject suffering of a ciliopathy, wherein the polyribonucleotide encodes a functional version of a protein a defect of which is associated with said ciliopathy, and wherein administration of said pharmaceutical composition to the respiratory system of said subject is effected when the subject shows an inflammation of the respiratory system. Further, the present disclosure relates to a method for analyzing the effect of a polyribonucleotide on ciliogenesis, wherein said polyribonucleotide encodes a protein involved in and/or required for ciliogenesis.
MX2021009388A 2019-02-14 2020-02-13 Treatment of ciliopathies. MX2021009388A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP19157210 2019-02-14
PCT/EP2020/053774 WO2020165352A1 (en) 2019-02-14 2020-02-13 Treatment of ciliopathies

Publications (1)

Publication Number Publication Date
MX2021009388A true MX2021009388A (en) 2021-09-10

Family

ID=65440876

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021009388A MX2021009388A (en) 2019-02-14 2020-02-13 Treatment of ciliopathies.

Country Status (14)

Country Link
US (1) US20220211807A1 (en)
EP (2) EP4223306A3 (en)
JP (2) JP2022522412A (en)
KR (1) KR20210127718A (en)
CN (1) CN113423418A (en)
AU (1) AU2020220636A1 (en)
BR (1) BR112021014528A2 (en)
CA (2) CA3129912A1 (en)
ES (1) ES2974368T3 (en)
HU (1) HUE065017T2 (en)
MX (1) MX2021009388A (en)
PL (1) PL3923971T3 (en)
WO (1) WO2020165352A1 (en)
ZA (1) ZA202106769B (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3463483T3 (en) 2016-05-27 2024-03-04 Transcriptx Inc Treatment of primary ciliary dyskinesia with synthetic messenger RNA
US20240123087A1 (en) * 2021-03-19 2024-04-18 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
EP4313002A1 (en) * 2021-03-22 2024-02-07 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
US20240337645A1 (en) * 2021-08-04 2024-10-10 The Trustees Of Indiana University Methods of treating compromised lung function and assessing clinical improvement

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
ES2167939T3 (en) 1997-08-13 2002-05-16 Biontex Lab Gmbh NEW POLYAMINS, ITS PREPARATION AND USES.
ES2219346T3 (en) 1999-06-25 2004-12-01 Christian Plank COMBINATIONS FOR THE INTRODUCTION OF NUCLEIC ACIDS IN CELLS.
WO2005121348A1 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
FR2925491B1 (en) 2007-12-19 2010-09-03 Oz Biosciences Sas NEW CLASSES OF CATIONIC LIPIDS FOR THE TRANSPORT OF ACTIVE AGENTS IN CELLS
EA201492055A1 (en) * 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. INHALATIVE DELIVERY OF mRNA IN TIGHTNESS CELL TARGETS
EP3800254A1 (en) * 2012-06-08 2021-04-07 Ethris GmbH Pulmonary delivery of messenger rna
WO2014020723A1 (en) 2012-08-01 2014-02-06 株式会社コナミデジタルエンタテインメント Processing device, method for controlling processing device, and processing device program
US12064484B2 (en) * 2013-06-28 2024-08-20 Ethris Gmbh Compositions for introducing RNA into cells
KR102647743B1 (en) * 2014-11-10 2024-03-14 에트리스 게엠베하 Induction of osteogenesis by delivering bmp encoding rna
DK3463483T3 (en) * 2016-05-27 2024-03-04 Transcriptx Inc Treatment of primary ciliary dyskinesia with synthetic messenger RNA
GB201707212D0 (en) * 2017-05-05 2017-06-21 Ucl Business Plc Gene therapy for ciliopathies

Also Published As

Publication number Publication date
EP3923971C0 (en) 2023-12-20
ZA202106769B (en) 2024-01-31
WO2020165352A1 (en) 2020-08-20
HUE065017T2 (en) 2024-04-28
CN113423418A (en) 2021-09-21
CA3129912A1 (en) 2020-08-20
BR112021014528A2 (en) 2021-10-13
EP3923971B1 (en) 2023-12-20
JP2024096766A (en) 2024-07-17
KR20210127718A (en) 2021-10-22
ES2974368T3 (en) 2024-06-27
CA3225057A1 (en) 2020-08-20
AU2020220636A1 (en) 2021-07-29
EP3923971A1 (en) 2021-12-22
PL3923971T3 (en) 2024-03-25
EP4223306A3 (en) 2023-11-29
JP2022522412A (en) 2022-04-19
EP4223306A2 (en) 2023-08-09
US20220211807A1 (en) 2022-07-07

Similar Documents

Publication Publication Date Title
ZA202106769B (en) Treatment of ciliopathies
MX2020010689A (en) Defibrotide for the prevention and treatment of cytokine release syndrome and neurotoxicity associated with immunodepletion.
WO2019006005A3 (en) Methods and compositions for treating melanoma
MX2019011117A (en) B7-h3 antibody, antigen-binding fragment thereof and medical use thereof.
MX2021001807A (en) Combination of histone deacetylase inhibitor and protein kinase inhibitor and pharmaceutical use thereof.
EA201890891A1 (en) BIOMARKERS ASSOCIATED WITH INTERLEYKIN-33 DISEASES, AND THEIR APPLICATION
MX2017011486A (en) Methods of detecting and quantifying il-13 and uses in diagnosing and treating th2-associated diseases.
WO2020186132A8 (en) Plasma kallikrein inhibitors and uses thereof for treating hereditary angioedema attack
BR112021019262A2 (en) Use of cyclo-his-pro (chp) to prevent, mitigate or treat fibrosis
MX2019001633A (en) Silk-derived protein for treating inflammation.
AU2018282105A1 (en) Dosing of vibegron for treatment of overactive bladder
CR20210514A (en) Use of reboxetine to treat narcolepsy
BR112022007721A2 (en) METHOD FOR THE TREATMENT OF DEMENTIA
MX2023008115A (en) Chimeric receptor therapy.
BR112018014762A2 (en) method of treating (early) alzheimer's disease
ZA202002107B (en) Use of specific sirna against protein s for the treatment of hemophilia
MX2018004041A (en) Treatment of bile acid disorders.
BR112019001130A2 (en) use of lactam and pharmaceutical composition
MX2022003396A (en) Use of dkk-1 inhibitors for treating cancer.
BR112022009881A2 (en) CASPASE 6 INHIBITORS AND USES THEREOF
PH12020550066A1 (en) METHOD FOR TREATING TNFa-RELATED DISEASE
MX2021009247A (en) Methods, systems, and kits for treating inflammatory disease targeting il18r1.
MX2024005430A (en) Anti-tau antibody compositions, dosage forms, and methods.
MX2020007222A (en) Minocycline for treating inflammatory skin conditions.
WO2019055776A3 (en) Compositions and methods for treatment of diseases involving cxcl1 function